Actively Recruiting
Study of LX107 Gene Therapy by Subretinal Injection in Patients with AIPL1 Mutation-Related Inherited Retinal Dystrophy
Led by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Updated on 2026-03-10
13
Participants Needed
1
Research Sites
285 weeks
Total Duration
On this page
Sponsors
S
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Lead Sponsor
I
Innostellar Biotherapeutics Co.,Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of a gene therapy drug called LX107 in patients with retinal dystrophy caused by mutations in the AIPL1 gene. This early phase 1 study focuses on patients aged 4 years and older who have a confirmed diagnosis of this inherited retinal condition. The goal is to explore how well the gene therapy works and how safe it is for these patients. Participants will receive a one-time subretinal injection of LX107 on Day 0. Only one eye, selected by the investigator, will be treated during the study. The treatment involves delivering the gene therapy directly under the retina to target the genetic cause of the disease. No other interventions or comparator groups are mentioned. Throughout the study, participants will be closely monitored for treatment-emergent adverse events over a period of 6 months to assess safety and tolerability. Researchers will evaluate visual acuity and other eye health parameters, and participants will adhere to long-term follow-up protocols. The study includes comprehensive screening and safety assessments before and after treatment to ensure careful observation of participant health.
CONDITIONS
Official Title
A Study of LX107 Gene Therapy in AIPL1-IRD Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- The participant or guardian signs informed consent and agrees to long-term follow-up
- Age 4 years or older, adult or pediatric patients
- Confirmed diagnosis of inherited retinal dystrophy caused by biallelic AIPL1 gene mutations
- The study eye has a best-corrected visual acuity of no more than 58 letters (about 0.3 decimal) on the ETDRS chart at baseline
You will not qualify if you...
- History of eye diseases that could interfere with treatment or study results (e.g., glaucoma, diabetic retinopathy, retinal vein occlusion, retinal detachment, uveitis)
- Previous gene therapy for inherited retinal dystrophy or related hereditary eye diseases
- Insufficient viable retinal cells in the study eye
- Active infection in or around the study eye
- History of intraocular surgery within 6 months before screening
- Uncontrolled high blood pressure (systolic 60 160 mmHg or diastolic 60 100 mmHg)
- Diabetes with macrovascular complications, HbA1c over 7.5%, or treatment with multiple diabetes medications
- Other diseases or conditions judged by the investigator to pose risks or interfere with study participation
- Certain lab abnormalities including low platelet count, low hemoglobin, high liver enzymes, or impaired kidney function
- Use of other investigational drugs within 3 months before screening
- Unwillingness to use contraception or pregnancy/lactation during the study period
AI-Screening
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Trial Site Locations
Total: 1 location
1
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, China
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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